FDA Approves SpringWorks' GOMEKLI™ for NF1-PN Treatment

On February 11, 2025, SpringWorks Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved GOMEKLI™ (mirdametinib), a MEK inhibitor developed by the company, for treating adult and pediatric patients aged two years and above with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas (PN) that cannot be completely removed through surgery. This approval marks the first and only medicine sanctioned for both children and adults with NF1-PN. Along with the approval, SpringWorks received a rare pediatric disease priority review voucher from the FDA.

Saqib Islam, the Chief Executive Officer at SpringWorks, expressed gratitude for the opportunity to provide a treatment for NF1-PN patients, highlighting the importance of delivering a therapy that can shrink tumors and alleviate symptoms. He acknowledged the contributions of clinical trial participants, their families, investigators, and advocacy groups in making GOMEKLI accessible in the U.S.

Neurofibromatosis type 1 (NF1) is a genetic condition affecting around 100,000 individuals in the United States. The disorder predisposes patients to a 30-50% lifetime risk of developing plexiform neurofibromas—tumors that grow along the peripheral nerve sheath, potentially leading to severe disfigurement, pain, and functional impairment. Approximately 40,000 people in the U.S. live with NF1-PN, most of whom are adults lacking an approved medication until now. These tumors can evolve into malignant peripheral nerve sheath tumors, a severe and potentially fatal condition. Due to their infiltrative growth pattern, surgical removal of plexiform neurofibromas is often challenging and complete resection is not feasible in about 85% of cases.

Christopher Moertel, M.D., a lead investigator in the ReNeu trial, highlighted the challenges faced by NF1-PN patients and emphasized the significance of GOMEKLI in providing deep and durable responses with a manageable safety profile. He noted that the approval represents significant progress, particularly for adults who previously lacked an approved treatment option.

The FDA approval is grounded in findings from the Phase 2b ReNeu trial, which included 114 patients aged two years and above with NF1-PN. The trial demonstrated a 41% objective response rate (ORR) in adults and 52% in children. Tumor volume reductions were both deep and durable. In adults, the median best percentage change in target PN volume was -41%, and in children, it was -42%. Responses lasted at least 12 months in 88% of adults and 90% of children, with about half maintaining their response for at least 24 months. The trial also reported significant early and sustained improvements in pain and quality of life for patients.

The safety profile of GOMEKLI was found to be manageable, with the most common adverse events among adults being rash, diarrhea, nausea, musculoskeletal pain, vomiting, and fatigue. In children, common adverse events included rash, diarrhea, musculoskeletal pain, abdominal pain, vomiting, headache, and nausea.

Annette Bakker, Ph.D., CEO of the Children’s Tumor Foundation, commended the collaborative efforts that led to this approval, emphasizing the role of partnerships in advancing scientific innovations for treatments that may not have otherwise progressed. Kim Bischoff, Executive Director of NF Network, described NF1-PN as a complex disease impacting not only patients but their families, and welcomed the approval as a beacon of hope for improved outcomes.

SpringWorks is dedicated to aiding NF1-PN patients in accessing GOMEKLI through their CareConnections™ program, which offers personalized support services and resources, including insurance and financial assistance. GOMEKLI will be available in 1 and 2 mg capsules, as well as a 1mg tablet for oral suspension, and is expected to be distributed through a specialty pharmacy network in the U.S. within two weeks. The company is also seeking approval from the European Medicines Agency, with a decision anticipated in 2025.